Contributions
Abstract: PB2474
Type: Publication Only
Background
Pure red cell aplasia (PRCA) occurs in 7,5% to 16% of AB0 major mismatched allogeneic HSCT recipients. Several treatments have been applied but their results are largely variable.
Aims
To describe the use of eltrombopag for the treatment of refractory PRCA after HSCT.
Methods
We describe two PRCA patients resistant to multiple therapeutic options, treated with thrombopoietin mimetic eltrombopag (used off label) at the dose of 75 mg qd for the first two weeks and then increased to 150 mg qd. Both patients received a major ABO-incompatible HSCT (donor A pos, recipient O pos) after myeloablative conditioning regimen. In both cases RBC reduction of the apheretic stem cell product was not performed due to the low hematocrit value.
Results
Patient 1: A 48-year old male with high risk acute myeloid leukemia underwent matched unrelated donor (MUD) HSCT. Pre-transplant anti-A isohemoagglutinin titer was 1:512. Neutrophil engraftment occurred on day + 16; subsequently the patient developed transfusion dependent anemia, and on day +100 the bone marrow biopsy documented the presence of a PRCA with full donor chimerism. The patient was treated with theta-erythropoietin (rHuEPO) 40000 U per week and subsequently received 5 plasma exchange (PEX) procedures without significant response; from day + 234, 4 weekly doses of Rituximab 375 mg/m2/week were administered without any beneficial effect. At 6 months post-HSCT, iron chelation therapy (ICT) with deferasirox (DFX) was started and discontinued after 3 months due to grade III neutropenia. At 14 months post-HSCT, eltrombopag (ETP) was started due to persistent transfusion dependent anemia along with mild leukopenia and thrombocytopenia, leading to rapid improvement of blood cell counts. After 4 months of treatment, ETP was discontinued with an haemoglobin value of 15,4 gr/dl. At the present, the patient is off treatment since 6 months with normal haemoglobin levels and neutrophil counts. The bone marrow biopsy shows a normal cellularity (80%) with good trilinear representation and no evidence of clonal evolution.
Patient 2: A 47-year old female with biphenotypic acute leukemia received a MUD HSCT. Patient’s anti-A isohemoagglutitin pre transplant titer was 1:256. After engraftment, on day +47 treatment with rHuEPO 40000 U per week was started due to transfusion dependent anemia, with no remarkable response. A bone marrow biopsy on day + 100 was consistent with the diagnosis of PRCA. Chimerism was full donor. PEX procedures were discontinued due to a severe anaphylactic reaction to plasmatic proteins. Rituximab (375 mg/sqm/week for 4 weeks) and 4 doses of Bortezomib (1,4 mg/mq once a week) followed by one dose of Cyclophosphamide (1 gr) were administered without any significant response. At 5 months post HSCT ICT with DFX was started, but stopped after six months due to neutropenia, and replaced by DFO. At 30 months post transplant ETP was started. After 5 weeks the patient became transfusion independent. ETP dose was reduced due to mild thrombocytosis. After 5 months of treatment the bone marrow biopsy showed a moderate trilinear hyperplasia with no evidence of clonal evolution. After 6 months of treatment, ETP was stopped, and the patient mantains normal blood cell counts.
Conclusion
Our results, even if restricted to a very small proportion of patients, may be considered indicative of a favorable effect of ETP on post transplant unilineage cytopenias such as PRCA. Additional studies are mandatory to confirm our preliminary data.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Allogeneic hematopoietic stem cell transplant, Pure red cell aplasia
Abstract: PB2474
Type: Publication Only
Background
Pure red cell aplasia (PRCA) occurs in 7,5% to 16% of AB0 major mismatched allogeneic HSCT recipients. Several treatments have been applied but their results are largely variable.
Aims
To describe the use of eltrombopag for the treatment of refractory PRCA after HSCT.
Methods
We describe two PRCA patients resistant to multiple therapeutic options, treated with thrombopoietin mimetic eltrombopag (used off label) at the dose of 75 mg qd for the first two weeks and then increased to 150 mg qd. Both patients received a major ABO-incompatible HSCT (donor A pos, recipient O pos) after myeloablative conditioning regimen. In both cases RBC reduction of the apheretic stem cell product was not performed due to the low hematocrit value.
Results
Patient 1: A 48-year old male with high risk acute myeloid leukemia underwent matched unrelated donor (MUD) HSCT. Pre-transplant anti-A isohemoagglutinin titer was 1:512. Neutrophil engraftment occurred on day + 16; subsequently the patient developed transfusion dependent anemia, and on day +100 the bone marrow biopsy documented the presence of a PRCA with full donor chimerism. The patient was treated with theta-erythropoietin (rHuEPO) 40000 U per week and subsequently received 5 plasma exchange (PEX) procedures without significant response; from day + 234, 4 weekly doses of Rituximab 375 mg/m2/week were administered without any beneficial effect. At 6 months post-HSCT, iron chelation therapy (ICT) with deferasirox (DFX) was started and discontinued after 3 months due to grade III neutropenia. At 14 months post-HSCT, eltrombopag (ETP) was started due to persistent transfusion dependent anemia along with mild leukopenia and thrombocytopenia, leading to rapid improvement of blood cell counts. After 4 months of treatment, ETP was discontinued with an haemoglobin value of 15,4 gr/dl. At the present, the patient is off treatment since 6 months with normal haemoglobin levels and neutrophil counts. The bone marrow biopsy shows a normal cellularity (80%) with good trilinear representation and no evidence of clonal evolution.
Patient 2: A 47-year old female with biphenotypic acute leukemia received a MUD HSCT. Patient’s anti-A isohemoagglutitin pre transplant titer was 1:256. After engraftment, on day +47 treatment with rHuEPO 40000 U per week was started due to transfusion dependent anemia, with no remarkable response. A bone marrow biopsy on day + 100 was consistent with the diagnosis of PRCA. Chimerism was full donor. PEX procedures were discontinued due to a severe anaphylactic reaction to plasmatic proteins. Rituximab (375 mg/sqm/week for 4 weeks) and 4 doses of Bortezomib (1,4 mg/mq once a week) followed by one dose of Cyclophosphamide (1 gr) were administered without any significant response. At 5 months post HSCT ICT with DFX was started, but stopped after six months due to neutropenia, and replaced by DFO. At 30 months post transplant ETP was started. After 5 weeks the patient became transfusion independent. ETP dose was reduced due to mild thrombocytosis. After 5 months of treatment the bone marrow biopsy showed a moderate trilinear hyperplasia with no evidence of clonal evolution. After 6 months of treatment, ETP was stopped, and the patient mantains normal blood cell counts.
Conclusion
Our results, even if restricted to a very small proportion of patients, may be considered indicative of a favorable effect of ETP on post transplant unilineage cytopenias such as PRCA. Additional studies are mandatory to confirm our preliminary data.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Allogeneic hematopoietic stem cell transplant, Pure red cell aplasia